SEL-212

WALTHAM, Mass., Nov. 07, 2024 (GLOBE NEWSWIRE) -- Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®), today announced the presentation of four abstracts that highlights data from its immunology portfolio at the ACR Convergence 2024 taking place in Washington, D.C. from November 14–19, 2024. Sobi’s commitment to delivering innovative treatments for people living with immunological diseases is seen in global studies spanning multiple disorders: Results from two Phase 3 trials evaluating the effect on patient-reported outcomes in patients with chronic refractory gout treated with SEL-212.Initial data on the effect of anti-drug antibodies on serum uric acid (sUA) reduction and safety in response to treatment with SEL-212.DISSOLVE 1 Phase 3 study: Results of a post hoc analysis of sustained sUA reduction and safety of SEL-212 in patients with chronic refractory gout living in the United States.Results from a Phase 3 Study and a pooled analysis of two prospective trials evaluating efficacy and safety of emapalumab in patients with Macrophage Activation Syndrome in Still’s disease. Key Sobi data to be presented at ACR Convergence 2024 Chronic Refractory GoutSEL-212Improvements in Patient-Reported Outcomes After Treatment with SEL-212 in Adults with Refractory Gout: Results from Two Randomized Phase 3 TrialsSession: Patient Outcomes, Preferences, & Attitudes Poster ISaturday, November 16; 10:30 AM - 12:30 PMImpact of Anti-drug Antibodies on the Efficacy of SEL-212 in Patients with Chronic Gout Refractory to Conventional TherapySafety and Efficacy of SEL-212 in the US and ex-US Subgroups: Results from the Phase 3 DISSOLVE StudiesSession: Metabolic & Crystal Arthropathies – Basic & Clinical Science Poster IIIMonday, November 18; 10:30 AM - 12:30 PM Macrophage Activation SyndromeEmapalumabEfficacy and Safety of Emapalumab in Children and Adults with Macrophage Activation Syndrome (MAS) in Still’s disease: Results from a Phase 3 Study and a Pooled Analysis of Two Prospective TrialsLate-Breaking AbstractsNovember 19; Session Time: 8:00 AM - 9:30 AM; Presentation Time: 9:00 AM - 9:15 AM All abstracts can be accessed via the official ACR Convergence website. ContactsFor details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media, click here. About SEL-212SEL-212 is being developed as a novel once-monthly combination medicine that integrates targeted immunomodulation with uricase-based therapy and is being investigated for the treatment of chronic refractory gout . SEL-212 consists of pegadricase, a proprietary pegylated uricase, co-administered with nanoencapsulated sirolimus. About Chronic Refractory GoutGout is the most common form of inflammatory arthritis with more than 8.3 million people in the United States having been diagnosed with gout, which is caused by high levels of uric acid in the body that accumulate around the joints and other tissues and can result in flares that cause intense pain. Approximately 160,000 people in the United States suffer from chronic refractory gout (CRG), where patients are resistant to conventional medicines and symptoms are more persistent. CRG is a painful and debilitating condition in people with persistent raised serum uric acid (sUA) levels, which may result in several flares per year and the development of nodular masses of uric acid crystals known as tophi. About emapalumab-lzsgEmapalumab-lzsg is a fully human, anti-IFNγ monoclonal antibody that binds free and receptor-bound IFNγ, neutralizing its biological activity. In the US, emapalumab-lzsg is indicated for pediatric (newborn and older) and adult primary hemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy. Emapalumab-lzsg is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval in 2018 was based on data from the phase 2/3 studies (NCT01818492 and NCT02069899).For the treatment of HLH, Emapalumab is indicated for administration through twice per week until hematopoietic stem cell transplantation (HSCT). The efficacy and safety of emapalumab are currently being evaluated. Emapalumab is currently under investigation (phase 3) in patients with MAS in Still’s disease or systemic lupus erythematosus (SLE) (EMERALD; NCT05001737). U.S. Indication for Gamifant® (emapalumab-lzsg)Gamifant® (emapalumab-lzsg) is an interferon gamma (IFNγ)–blocking antibody indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy. Important Safety InformationInfectionsBefore initiating Gamifant, patients should be evaluated for infection, including latent tuberculosis (TB). Prophylaxis for TB should be administered to patients who are at risk for TB or known to have a positive purified protein derivative (PPD) test result or positive IFNγ release assay. During Gamifant treatment, patients should be monitored for TB, adenovirus, Epstein-Barr virus (EBV), and cytomegalovirus (CMV) every 2 weeks and as clinically indicated. Patients should be administered prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infections prior to Gamifant administration. Increased Risk of Infection With Use of Live VaccinesDo not administer live or live attenuated vaccines to patients receiving Gamifant and for at least 4 weeks after the last dose of Gamifant. The safety of immunization with live vaccines during or following Gamifant therapy has not been studied. Infusion-Related ReactionsInfusion-related reactions, including drug eruption, pyrexia, rash, erythema, and hyperhidrosis, were reported with Gamifant treatment in 27% of patients. In one-third of these patients, the infusion-related reaction occurred during the first infusion. Adverse ReactionsIn the pivotal trial, the most commonly reported adverse reactions (≥10%) for Gamifant included infection (56%), hypertension (41%), infusion-related reactions (27%), pyrexia (24%), hypokalemia (15%), constipation (15%), rash (12%), abdominal pain (12%), CMV infection (12%), diarrhea (12%), lymphocytosis (12%), cough (12%), irritability (12%), tachycardia (12%), and tachypnea (12%). Additional selected adverse reactions (all grades) that were reported in less than 10% of patients treated with Gamifant included vomiting, acute kidney injury, asthenia, bradycardia, dyspnea, gastrointestinal hemorrhage, epistaxis, and peripheral edema. Please see the full Prescribing Information for Gamifant. About Sobi North AmericaAs the North American affiliate of international biopharmaceutical company Sobi, the Sobi North America team is committed to Sobi’s vision of being a leader in providing innovative treatments that transform lives for individuals with rare diseases. Our product portfolio includes multiple approved treatments focused on immunology, hematology and specialty care. With U.S. headquarters in the Boston area, Canadian headquarters in the Toronto area, and field sales, medical and market access representatives spanning North America, our growing team has a proven track record of commercial excellence. More information is available at https://www.sobi.com/usa or at www.sobi.com. About Sobi®Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of hematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube. Brian Castellibrian.castelli@sobi.com

Presented positive topline results from Phase 2b trial of Descartes-08 in patients with myasthenia gravis; End-of-Phase 2 meeting with FDA expected by year-end Dosed first SLE patient in Phase 2 trial of Descartes-08 IND filing for pediatric basket study of Descartes-08 with focus in neurology and rheumatology expected by year-end PIPE financing strengthened balance sheet, with net proceeds expected to support development of Descartes-08 in MG through planned Phase 3 trial GAITHERSBURG, Md., Aug. 08, 2024 (GLOBE NEWSWIRE) -- Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (the “Company”), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, today reported financial results for the second quarter of 2024, and provided recent business and corporate updates. “Last quarter marked a pivotal milestone in Cartesian’s history as we demonstrated clinical differentiation of our novel mRNA platform,” said Carsten Brunn, Ph.D., President and Chief Executive Officer of Cartesian. “MG patients treated with Descartes-08 were observed to have deep and durable responses, supporting the potential breadth and application of Cartesian’s approach to treating autoimmune diseases. Additionally, we raised approximately $130 million from both new and existing investors to help us execute the planned Phase 3 trial of Descartes-08 in MG. We look forward to continuing our strong momentum, meeting with the FDA before year-end and initiating a Phase 3 clinical trial in MG, filing an IND for a pediatric basket study, and expanding our pipeline to address new disease indications.” Recent Pipeline Progress and Anticipated Milestones Descartes-08 for Myasthenia Gravis (MG) In July 2024, the Company presented positive topline results from its Phase 2b trial of Descartes-08 in patients with generalized MG. The trial achieved its primary endpoint with statistical significance in the pre-specified modified intent-to-treat efficacy population, with 71% (10/14) of patients treated with Descartes-08 observed to have 5-point or greater improvements in MG Composite (MGC) score at Month 3 compared to 25% (3/12) of patients treated with placebo (p=0.018).Responders that reached their four-month and six-month assessments were observed to have deep, durable, and clinically meaningful improvements in their MGC severity scores.Descartes-08 was observed to have a favorable safety profile supporting outpatient administration without the need for lymphodepleting chemotherapy. The Company expects to hold an End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) by year-end 2024 to review data from the Phase 2b trial and discuss plans for initiating a Phase 3 clinical trial of Descartes-08 in MG. Descartes-08 was previously granted Regenerative Medicine Advanced Therapy (RMAT) Designation, which allows for more frequent regulatory engagement, and Orphan Drug Designation by the FDA for the treatment of MG.Descartes-08, the Company’s lead product candidate, is an autologous anti-B cell maturation antigen (BCMA) mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T). Descartes-08 for Systemic Lupus Erythematosus (SLE) In July 2024, the Company announced dosing of the first patient in a Phase 2 clinical trial.The trial is designed to assess the safety, tolerability and clinical activity of outpatient Descartes-08 administration without preconditioning chemotherapy in patients with SLE.The Company believes that the mechanism of action of Descartes-08, which targets both plasma cells and plasmacytoid dendritic cells, could lead to clinical benefit in patients with SLE.SLE is an incurable autoimmune disease marked by systemic inflammation that affects multiple organ systems and impacts approximately 1.5 million people in the United States. Descartes-08 for Pediatric Autoimmune Diseases Cartesian plans to file an Investigational New Drug (IND) application for Descartes-08 in pediatric autoimmune disease indications by year-end 2024.The planned basket trial will focus on certain pediatric neurological and rheumatological autoimmune diseases that have high unmet medical need.To date, Descartes-08 has been observed to have a favorable safety profile in adult patients treated in an outpatient setting without lymphodepleting chemotherapy, which the Company believes could be a key differentiator for treating pediatric patients. Descartes-15 for Autoimmune Diseases The Company expects to dose the first patient in its planned Phase 1 trial of Descartes-15 in the second half of 2024.The Phase 1 dose escalation trial will assess the safety and tolerability of outpatient Descartes-15 administration in patients with multiple myeloma. Following the Phase 1 dose escalation trial, the Company expects to subsequently assess Descartes-15 in autoimmune indications.Descartes-15 is a next-generation autologous anti-BCMA mRNA CAR-T product candidate designed to have predictable and controllable pharmacokinetics, including technological advances that enhance CAR stability even in the presence of target-driven suppression of CAR.Similar to Descartes-08, Descartes-15 is designed to be administered without preconditioning chemotherapy and eliminate integrating vectors.Relative to Descartes-08, Descartes-15 has been observed to achieve an approximately ten-fold increase in CAR expression and selective target-specific killing in preclinical studies. Corporate Updates Completed $130 Million Private Placement Equity Financing In July 2024, Cartesian announced a private investment in public equity (PIPE) financing, which included participation from both new and existing investors, resulting in gross proceeds of approximately $130.0 million.The Company intends to use the net proceeds from the PIPE financing, together with the Company’s existing cash, cash equivalents, and restricted cash, to continue development of Descartes-08 in MG, specifically supporting anticipated manufacturing costs associated with a Phase 3 clinical trial and early commercial activities in preparation for a potential launch, if approved.Additionally, Cartesian expects to use the net proceeds to advance and expand its autoimmune pipeline through continued development of Descartes-08 for SLE, Descartes-15 for autoimmune diseases, and prepare for a planned basket trial for autoimmune pediatric indications.Operationally, the Company expects to continue making enhancements to its process development and manufacturing capabilities to improve production yields. Strengthened Board of Directors with Appointment of Kemal Malik In July 2024, the Company announced the appointment of Kemal Malik, MBBS to its Board of Directors.Dr. Malik’s appointment provides regulatory and clinical expertise and deepens the Company’s strategic leadership. He has over 30 years of global development, regulatory, and commercial experience at leading pharmaceutical organizations. Second Quarter 2024 Financial Results Cash, cash equivalents, and restricted cash were approximately $88.9 million as of June 30, 2024. In conjunction with net proceeds from the $130.0 million PIPE financing announced in July 2024, the Company’s cash, cash equivalents, and restricted cash as of June 30, 2024 are expected to support development of Descartes-08 in MG, specifically supporting anticipated manufacturing costs associated with a Phase 3 clinical trial and early commercial activities in preparation for a potential launch, and help support the advancement and expansion of its autoimmune pipeline, including Descartes-08 for SLE, other potential indications, and enhancements to its process development and manufacturing capabilities.Research and development expenses were $12.7 million for the quarter ended June 30, 2024, compared to $17.8 million for the quarter ended June 30, 2023. The decrease in research and development expenses of $5.1 million for the quarter ended June 30, 2024 was due to a one-time cash charge to salaries and benefits as a result of headcount reduction in April 2023 and decreased contract license and milestone payments.General and administrative expenses were $7.0 million for the quarter ended June 30, 2024, compared to $6.1 million for the quarter ended June 30, 2023. The increase in general and administrative expenses of $0.9 million for the quarter ended June 30, 2024 was primarily due to personnel expenses.Net income was $13.8 million, or basic net income per share allocable to common stockholders of $0.58, for the quarter ended June 30, 2024, compared to net loss of $(11.4) million, or basic net loss per share allocable to common stockholders of $(2.23), for the quarter ended June 30, 2023. The net income includes recognition of revenue for a $30.0 million milestone fee, which was triggered by the initiation of a Biologics License Applications filing for SEL-212 by Swedish Orphan Biovitrum AB (Sobi). The milestone payment is expected to be paid out to Contingent Value Rights (CVR) holders in March 2025 net of deductions specified in the CVR Agreement. About Descartes-08 Descartes-08, Cartesian’s lead mRNA cell therapy candidate and a potential first-in-class mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T), is an autologous mRNA CAR-T product targeting B-cell maturation antigen (BCMA) in clinical development for generalized myasthenia gravis (MG) and systemic lupus erythematosus. In contrast to conventional DNA-based CAR T-cell therapies, mRNA CAR-T administration does not require preconditioning chemotherapy, can be administered in the outpatient setting, and does not carry the risk of genomic integration associated with cancerous transformation. Descartes-08 has been granted Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation by the U.S. Food and Drug Administration for the treatment of MG. About Cartesian Therapeutics Cartesian Therapeutics is a clinical-stage company pioneering mRNA cell therapies for the treatment of autoimmune diseases. The Company’s lead asset, Descartes-08, is a potential first-in-class mRNA CAR-T in Phase 2b clinical development for patients with generalized myasthenia gravis and Phase 2 development for systematic lupus erythematosus, with a Phase 2 basket trial planned in additional autoimmune indications. The Company’s clinical-stage pipeline also includes Descartes-15, a next-generation, autologous anti-BCMA mRNA CAR-T. For more information, please visit www.cartesiantherapeutics.com or follow the Company on LinkedIn or X, formerly known as Twitter. Forward Looking Statements Any statements in this press release about the future expectations, plans and prospects of the Company, including without limitation, statements regarding the Company’s expectation to hold an End-of-Phase 2 meeting with the FDA by the end of 2024, the ability of Descartes-08 to be administered in an outpatient setting or without the need for preconditioning lymphodepleting chemotherapy, the Company’s in-house manufacturing capabilities, the potential of the Company’s technology to enable precision control and optimization of engineered cells for diverse cell therapies leveraging multiple modalities, the potential of Descartes-08, Descartes-15, or any of the Company’s other product candidates to treat myasthenia gravis, systemic lupus erythematosus, or any other disease, the amount and occurrence of any payments to holders of the Company’s contingent value rights, the anticipated timing or the outcome of ongoing and planned clinical trials, studies and data readouts, the anticipated timing or the outcome of the FDA’s review of the Company’s regulatory filings, the Company’s ability to conduct its clinical trials and preclinical studies, the timing or making of any regulatory filings, the anticipated timing or outcome of selection of developmental product candidates, the ability of the Company to consummate any expected agreements and licenses and to realize the anticipated benefits thereof, the novelty of treatment paradigms that the Company is able to develop, the potential of any therapies developed by the Company to fulfill unmet medical needs, and enrollment in the Company’s clinical trials and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “hypothesize,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial and whether results of early clinical trials will be indicative of the results of later clinical trials, the ability to predict results of studies performed on human beings based on results of studies performed on non-human subjects, the unproven approach of the Company’s technology, potential delays in enrollment of patients, undesirable side effects of the Company’s product candidates, its reliance on third parties to conduct its clinical trials, the Company’s inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the Company’s recurring losses from operations and negative cash flows, substantial fluctuation in the price of the Company’s common stock, risks related to geopolitical conflicts and pandemics and other important factors discussed in the “Risk Factors” section of the Company’s most recent Annual Report on Form 10-K and subsequently filed Quarterly Reports on Form 10-Q, and in other filings that the Company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the Company’s views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The Company specifically disclaims any intention to update any forward-looking statements included in this press release, except as required by law. Cartesian Therapeutics, Inc. and SubsidiariesConsolidated Balance Sheets(Amounts in thousands, except share data and par value) June 30, 2024 December 31, 2023 (Unaudited) Assets Current assets: Cash and cash equivalents$87,227 $76,911Accounts receivable 32,039 5,870Unbilled receivables 3,472 2,981Prepaid expenses and other current assets 2,044 4,967Total current assets 124,782 90,729Non-current assets: Property and equipment, net 6,672 2,113Right-of-use asset, net 13,852 10,068In-process research and development assets 150,600 150,600Goodwill 48,163 48,163Long-term restricted cash 1,669 1,377Investments 2,000 2,000Total assets$347,738 $305,050Liabilities, convertible preferred stock, and stockholders’ deficit Current liabilities: Accounts payable$2,862 $3,150Accrued expenses and other current liabilities 10,954 15,572Lease liability 2,523 2,166Deferred revenue — 2,311Warrant liabilities 1,205 720Contingent value right liability 8,571 15,983Forward contract liabilities — 28,307Total current liabilities 26,115 68,209Non-current liabilities: Lease liability, net of current portion 12,344 8,789Deferred revenue, net of current portion — 3,538Warrant liabilities, net of current portion 8,055 5,674Contingent value right liability, net of current portion 386,829 342,617Deferred tax liabilities, net 15,853 15,853Total liabilities 449,196 444,680Series A Preferred Stock, $0.0001 par value; no and 548,375 shares authorized as of June 30, 2024 and December 31, 2023, respectively; no and 435,120.513 shares issued and outstanding as of June 30, 2024 and December 31, 2023, respectively — 296,851Options for Series A Preferred Stock — 3,703Stockholders’ deficit: Series A Preferred Stock, $0.0001 par value; 180,455.753 and no shares authorized as of June 30, 2024 and December 31, 2023, respectively; 166,341.592 and no shares issued and outstanding as of June 30, 2024 and December 31, 2023, respectively — —Preferred stock, $0.0001 par value; 9,819,544.247 and 9,451,625 shares authorized as of June 30, 2024 and December 31, 2023, respectively; no shares issued and outstanding as of June 30, 2024 and December 31, 2023 — —Common stock, $0.0001 par value; 350,000,000 shares authorized as of June 30, 2024 and December 31, 2023; 17,816,238 and 5,397,597 shares issued and outstanding as of June 30, 2024 and December 31, 2023, respectively 2 1Additional paid-in capital 560,766 179,062Accumulated deficit (657,635) (614,647)Accumulated other comprehensive loss (4,591) (4,600)Total stockholders’ deficit (101,458) (440,184)Total liabilities, convertible preferred stock, and stockholders’ deficit$347,738 $305,050 Cartesian Therapeutics, Inc. and SubsidiariesConsolidated Statements of Operations and Comprehensive Income (Loss)(Amounts in thousands, except share and per share data) Three Months EndedJune 30, Six Months EndedJune 30, 2024 2023 2024 2023 (Unaudited) Revenue: Collaboration and license revenue$33,271 $5,249 $39,111 $11,187 Grant revenue 174 — 174 — Total revenue 33,445 5,249 39,285 11,187 Operating expenses: Research and development 12,661 17,782 22,399 36,406 General and administrative 7,027 6,105 16,477 11,800 Total operating expenses 19,688 23,887 38,876 48,206 Operating income (loss) 13,757 (18,638) 409 (37,019)Investment income 1,195 1,394 2,359 2,725 Foreign currency transaction, net — 23 — 42 Interest expense — (752) — (1,560)Change in fair value of warrant liabilities (3,908) 6,341 (2,866) 2,262 Change in fair value of contingent value right liability 2,500 — (36,800) — Change in fair value of forward contract liabilities — — (6,890) — Other income, net 292 245 800 500 Net income (loss)$13,836 $(11,387) $(42,988) $(33,050) Other comprehensive income (loss): Foreign currency translation adjustment 14 (27) 9 (49)Unrealized gain on marketable securities — — — 11 Total comprehensive income (loss)$13,850 $(11,414) $(42,979) $(33,088) Net income (loss) per share allocable to common stockholders: Basic$0.58 $(2.23) $(3.88) $(6.46)Diluted$0.54 $(2.23) $(3.88) $(6.46)Weighted-average common shares outstanding: Basic 16,723,479 5,114,747 11,068,749 5,113,213 Diluted 17,791,143 5,114,747 11,068,749 5,113,213 Investor ContactRon MoldaverSenior Director, Investor Relations & Business Developmentron.moldaver@cartesiantx.com Media ContactDavid RosenArgot Partnersdavid.rosen@argotpartners.com